AveXis/Novartis received FDA approval for Zolgensma, the gene therapy for children younger than two years old who have been diagnosed with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. The therapy will be available to patients for a cost of $2.125 million, or $425,000 per year for 5 years. https://www.biospace.com/article/novartis-sma-gene-therapy-treatment-approved-by-the-fda-will-have-a-price-of-2-125-million/
Novocure said the FDA approved its NovoTTF-100L System in combination with pemetrexed plus platinum-based chemotherapy as a first-line treatment of unresectable, locally advanced or metastatic, malignant pleural mesothelioma. This marks the first FDA approval for malignant pleural mesothelioma (MPM) in 15 years. https://www.biospace.com/article/novocure-wins-fda-approval-for-first-mesothelioma-treatment-in-15-years/?utm
The FDA has approved Incyte Corporation’s Jakafi for treatment of steroid-refractory acute graft-versus-host disease in adult and pediatric patients 12 years and older. https://www.biospace.com/article/incyte-s-jakafi-snags-a-third-fda-approval-for-treatment-of-steroid-refractory-acute-gvhd/?utm
The FDA approved Novartis’s Piqray for breast cancer. This is the first NDA approved under the Real-Time Oncology Review pilot program (RTOR): https://www.raps.org/news-and-articles/news-articles/2019/5/fda-approves-first-nda-under-real-time-oncology-re?utm
Cadent Therapeutics announced that the FDA has granted Orphan Drug Designation to CAD-1883, an investigational treatment for spinocerebellar ataxia (SCA), a genetic disorder characterized by progressive loss of coordination, slurred speech, difficulty controlling eye movements and cognitive dysfunction. https://www.firstwordpharma.com/node/1643840
In other news…
Aegerion Pharmaceuticals has entered into a plan funding agreement and a restructuring support agreement that will result in Aegerion selling 100% of its reorganized stock to, and becoming a wholly-owned subsidiary of, Dublin-based Amryt Pharma Plc. https://finance.yahoo.com/news/novelion-therapeutics-announces-subsidiary-aegerion-025320504.html
AbbVie noted that depatuxizumab mafodotin (or depatux-m; previously known as ABT-414) has been failing to spark a survival benefit in EGFR-amplified cases of glioblastoma in a Phase III study, causing the independent monitors to call it off for the antibody-drug conjugate. https://endpts.com/abbvie-concedes-another-phiii-cancer-drug-defeat-as-glioblastoma-defeats-another-assault/
Dynavax said it needed to focus all its resources on its 2017-approved Heplisav-B and its broader vaccine business, so it is exploring strategic alternatives for its immuno-oncology programs and cutting jobs. https://endpts.com/sobered-by-mounting-costs-dynavax-seeks-strategic-alternatives-for-cancer-assets-and-cuts-jobs-to-focus-on-heplisav/
Stoke Therapeutics has set a preliminary $86 million target for its IPO to help bring its Dravel Syndrome drug into the clinic: https://xconomy.com/boston/2019/05/24/stoke-therapeutics-preps-ipo-to-bring-dravet-syndrome-drug-to-clinic/?mc_cid=fc6ee0a1b5&mc_eid=3e3fa57749
Eli Lilly announced it has inked a license agreement with Centrexion Therapeutics to acquire the exclusive global rights to CNTX-0290. The drug is a small molecule somatostatin receptor type 4 (SSTR4) agonist being evaluated in Phase I as a possible non-opioid therapy for chronic pain. https://www.biospace.com/article/eli-lilly-in-licenses-non-opioid-compound-from-centrexion/?utm
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